NORD, BioNJ and Patient Advocacy Groups to Collaborate on Rare Disease Day Event at New Jersey State House

TRENTON, NJ (February 13, 2013) — The National Organization for Rare Disorders, BioNJ and 11 New Jersey-based patient advocacy groups are joining forces for a unique educational event about rare diseases to be held at the New Jersey State House on March 4, 2013. The gathering, which will convene local leaders from government, non-profit, and the biotechnology sector, will be conducted on behalf of state residents living with rare diseases that plague nearly 1 in every 10 Americans. The event is highlighted by a New Jersey success story – the latest in-state biopharmaceutical company to offer a significant treatment advance for one group of patients with a rare intestinal disorder.

Patients, researchers, health care advocates, members of the New Jersey government and biotech industry, as well as concerned citizens are invited to attend the event on Monday, March 4, 2013, from 11:30 a.m. to 12:30 p.m. at Committee Room 11, 4th floor, Annex Building, New Jersey Statehouse.

A wide variety of speakers will include researchers, clinicians, patients, patient advocates and members of industry, including:

  • Francois Nader, MD, President and CEO of NPS Pharmaceuticals of Bedminster, NJ.  In December, NPS received approval from the U.S. Food and Drug Administration (FDA) for Gattex® (teduglutide [rDNA origin]) for injection as a treatment for adult patients with short bowel syndrome who are dependent on parenteral support.  Dr. Nader was elected Chairman of the BioNJ Board of Trustees earlier this month.
  • Debbie Hart, President and CEO of BioNJ
  • Mary Cobb, Senior Vice President, Membership at NORD
  • Ian Marshall, M.D., pediatric endocrinology specialist at Robert Wood Johnson University Hospital in New Brunswick, NJ.
  • Ellen Welch, Director of Biologics (Genetic Disorders) at PTC Therapeutics of Plainfield, NJ.
  • Diana Autin, Executive Co-Director of the Statewide Patient Advocacy Network (SPAN) of New Jersey

New Jersey State Senator Anthony Bucco (R-25) will also offer a resolution designating the last day of February as Rare Disease Day and honoring all New Jersey rare disease organizations.

The purpose of this event is to deliver the message that every patient counts and that all patients, even those suffering from rare disorders, need excellent health care and services to ensure a high quality of life. In the United States, a disease is considered rare if it affects fewer than 200,000 people.  There are more than 7,000 rare diseases affecting more than 30 million people in the U.S. alone.

This event is one of many being held in honor of Rare Disease Day, an international advocacy day bringing widespread recognition of rare diseases as a global health challenge.  NORD is the leader of rare disease activities in the U.S. and is coordinating events at state houses across the country.

In addition to NORD and BioNJ, participating patient organizations include:

  • Soft Bones
  • Congenital Hyperinsulinism International
  • CARES Foundation
  • NOMID Alliance
  • The Sturge Weber Foundation
  • APS Type 1
  • The Mastocytosis Society
  • Children’s Cardiomyopathy Foundation
  • “Let Them Be Little X2”
  • MDS Foundation